Trial reportThe Journal of clinical investigation2020
Sleeping Beauty-engineered CAR T cells achieve antileukemic activity without severe toxicities.
Trial report in The Journal of clinical investigation, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03389035 (Phase 1-2a Trial to Determine the Feasibility and Safety of a Single Dose of Transposon-manipulated Allogeneic CARCIK-CD19 Cells in Adult and Pediatric Patients With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia, After Hematopoietic Stem Cell Transplantation), which is not on this map. Cited by 132 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase 1-2a Trial to Determine the Feasibility and Safety of a Single Dose of Transposon-manipulated Allogeneic CARCIK-CD19 Cells in Adult and Pediatric Patients With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia, After Hematopoietic Stem Cell Transplantation
Who cites it
132 citing papers in PubMed, 2 syntheses or guidelines pooled it, 184 citations in OpenAlex.
- Incidence of immune effector cell-associated neurotoxicity among patients treated with CAR T-cell therapy for hematologic malignancies: systematic review and meta-analysis.Frontiers in neurology · 2024Pooled it
- Dose-response correlation for CAR-T cells: a systematic review of clinical studies.Journal for immunotherapy of cancer · 2022Pooled it
- Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation.Blood cancer journal · 2025Trial
- CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma.Gene therapy · 2021Trial
- A review of genetic modification for ex vivo cellular therapies.Transfusion · 2026Article
- Multiplex engineering of rhesus macaque NK cells enhances homing to sites of HIV replication in B cell follicles.bioRxiv : the preprint server for biology · 2026Article
- Are we there yet? The road to faster and more efficient CAR T cell manufacturing.Journal of biological engineering · 2026Review
- RAAVioli: A comprehensive approach to characterizing AAV vector integrations and rearrangements.Molecular therapy. Advances · 2026Article
- Unleashing CAR-T potential in solid tumors: overcoming intrinsic and extrinsic hurdles to improve therapy.Cancer immunology, immunotherapy : CII · 2026Review
- Characterization of Short Production Cycle and Nongenotoxic mRNA-Based CAR-T Cells Targeting CD19-Positive and CD22-Positive Malignancies.Journal of immunology research · 2026Article
- Development andFrontiers in immunology · 2026Article
- Therapeutic Potential of CAR-CIK Cells in Acute Leukemia Relapsed Post Allogeneic Stem Cell Transplantation.Cancers · 2025Review
- CAR T cell engineering impacts antigen-independent activation and co-inhibition.Molecular therapy. Methods & clinical development · 2025Article
- Breakthrough for Anticancer Immunotherapy: Current Advances in Manufacturing Protocols of Chimeric Antigen Receptor-Based Therapies.Antibodies (Basel, Switzerland) · 2025Review
- Next-generation T cell immunotherapies engineered with CRISPR base and prime editing: challenges and opportunities.Nature reviews. Clinical oncology · 2025Review
- Genome-edited allogeneic CAR-T cells: the next generation of cancer immunotherapies.Journal of hematology & oncology · 2025Review
- Acquisition of an immunosuppressive microenvironment after anti-CD19 CAR T-cell therapy is associated with T-cell dysfunction and resistance.Journal for immunotherapy of cancer · 2025Article
- Review
- Engineering of BZ transposase and transposon donor vector for enhanced efficiency and safety in gene delivery applications.Nucleic acids research · 2025Article
- Dual targeting of solid tumors using cytokine-induced killer cells modified with a CAR anti-tenascin C and a secretable EGFRxCD3 bispecific antibody.Cancer immunology, immunotherapy : CII · 2025Article
72 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
30 authors at 6 institutions in 1 country.
Funding
Abstract
BACKGROUNDChimeric antigen receptor (CAR) T cell immunotherapy has resulted in complete remission (CR) and durable response in highly refractory patients. However, logistical complexity and high costs of manufacturing autologous viral products limit CAR T cell availability.METHODSWe report the early results of a phase I/II trial in B cell acute lymphoblastic leukemia (B-ALL) patients relapsed after allogeneic hematopoietic stem cell transplantation (HSCT) using donor-derived CD19 CAR T cells generated with the Sleeping Beauty (SB) transposon and differentiated into cytokine-induced killer (CIK) cells.RESULTSThe cellular product was produced successfully for all patients from the donor peripheral blood (PB) and consisted mostly of CD3+ lymphocytes with 43% CAR expression. Four pediatric and 9 adult patients were infused with a single dose of CAR T cells. Toxicities reported were 2 grade I and 1 grade II cytokine-release syndrome (CRS) cases at the highest dose in the absence of graft-versus-host disease (GVHD), neurotoxicity, or dose-limiting toxicities. Six out of 7 patients receiving the highest doses achieved CR and CR with incomplete blood count recovery (CRi) at day 28. Five out of 6 patients in CR were also minimal residual disease negative (MRD-). Robust expansion was achieved in the majority of the patients. CAR T cells were measurable by transgene copy PCR up to 10 months. Integration site analysis showed a positive safety profile and highly polyclonal repertoire in vitro and at early time points after infusion.CONCLUSIONSB-engineered CAR T cells expand and persist in pediatric and adult B-ALL patients relapsed after HSCT. Antileukemic activity was achieved without severe toxicities.TRIAL REGISTRATIONClinicalTrials.gov NCT03389035.FUNDINGThis study was supported by grants from the Fondazione AIRC per la Ricerca sul Cancro (AIRC); Cancer Research UK (CRUK); the Fundación Científica de la Asociación Española Contra el Cáncer (FC AECC); Ministero Della Salute; Fondazione Regionale per la Ricerca Biomedica (FRRB).
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.